Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
mivelsiran
- Development stage
- Phase 2
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
Mivelsiran (ALN-APP) is an investigational RNA interference (RNAi) therapeutic designed to target and silence amyloid precursor protein (APP) messenger RNA in the central nervous system. By reducing APP mRNA, mivelsiran decreases synthesis of APP protein and all downstream cleavage products, including amyloid beta (Aβ), which are implicated in the pathogenesis of Alzheimer’s disease (AD) and cerebral amyloid angiopathy (CAA). The drug is administered intrathecally and utilizes Alnylam’s proprietary C16-siRNA conjugate technology for enhanced CNS delivery. Mivelsiran aims to reduce both intracellular and extracellular Aβ isoforms as well as other APP-derived peptides by acting upstream at the level of gene expression. It is currently being evaluated in clinical trials for CAA (Phase 2) and early-onset AD (Phase 1)[1][3][5][8].
- Other names
- trovacaptes
02
Targets
Aβ (Amyloid-beta peptides and aggregates)
Beyond the preview
Go deeper on mivelsiran.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on mivelsiran.